Efficacy of lenalidomide in POEMS syndrome: A retrospective study of 20 patients

Abstract

POEMS syndrome is a rare disorder characterized by polyneuropathy, monoclonal gammopathy, multiorgan involvement, and elevated vascular endothelial growth factor levels. Localized bone lesions require irradiation, whereas young patients with disseminated disease receive intensive treatment with stem cell support. Treatment of older and non responding patients is not yet standardized. We report the use of a combination of lenalidomide and dexamethasone in 20 patients with POEMS syndrome. Four patients were newly diagnosed, and 16 had relapsed or progressed after treatment. All but one of the patients responded: clinical improvements were noted in neuropathies (16/20) organomegaly (13/13), peripheral edema (14/15), and pulmonary hypertension (5/5). At least a very good partial response was noted in 68% of patients, with partial responses in 26%. Serum VEGF levels fell markedly in all 17 patients with available values. Twelve patients had 18‐FDG‐PET/CT at diagnosis (11 with positive findings), and nine patients during follow‐up. The number of lesions fell markedly in five cases and remained stable in two cases, while two patients became negative. During a median follow‐up of 22 months, four patients relapsed. Toxicity, predominantly hematological, was mild and manageable. Lenalidomide thus appears to be effective in POEMS syndrome, inducing high rate of clinical and biological responses. Am. J. Hematol. 88:207–212, 2013. © 2012 Wiley Periodicals, Inc.

POEMS syndrome: Update on diagnosis, risk‐stratification, and management

“…Although follow-up is short, the authors report that several patients had rapid neurological response, no patient has died, and one patient has progressed. These results are similar to previous case reports and case series [168,169,178] though relapses have been reported. In the largest case series of 20 patients [169], all patients responded, but 4 patients relapsed 3-10 months after the end of treatment.…”

“…These results are similar to previous case reports and case series [168,169,178] though relapses have been reported. In the largest case series of 20 patients [169], all patients responded, but 4 patients relapsed 3-10 months after the end of treatment. Three of these treatment failures responded to further therapy, including one who responded to reintroduction of the lenalidomide-dexamethasone combination.…”

Outcomes of patients with POEMS syndrome treated initially with radiation

“…1 Due to the rarity of the disease, prospective therapeutic clinical trials are rare. 2,3 Based on experience with patients with solitary plasmacytomas of bone (SPB), the approach for patients with POEMS, negative bone marrows, and limited bone lesions has been targeted irradiation for patients with 1 to 3 isolated bone lesions. 4 Anecdotal reports have shown benefit, [5][6][7][8][9][10] but there are no published data on long-term results or time to treatment failure with this approach.…”

High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation

“…11 More recently, two studies have demonstrated promising results in the phase II trial setting with favorable outcomes. [12][13][14] As with MM, the use of HDT/ASCT in patients with POEMS has yielded encouraging results, though most reported series are small and single center experiences. [15][16][17][18][19][20] We, therefore, sought to characterize the effect of ASCT as therapy for patients with POEMS syndrome, determining the effect on organ-specific dysfunction and delineating key patient-and disease-specific factors predicting for outcome.…”